UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
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FORM 8-K
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CURRENT REPORT
Pursuant to Section 13 OR 15 (d)
of The Securities Exchange Act of 1934
Date of Report (Date of Earliest Event Reported): March 13, 2023
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(Exact Name of Registrant as Specified in its Charter)
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| (State or Other Jurisdiction of Incorporation) | (Commission File Number) | (I.R.S. Employer Identification Number) | ||||||||||||
(484 ) 328-4701
(Address, including zip code, and telephone number, including area code, of principal executive office)
N/A
(Former Name or Former Address, if Changed Since Last Report)
Check the appropriate box below if the Form 8–K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||||||||||||
(The Nasdaq Capital Market) | ||||||||||||||
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 8.01 Other Events.
Attached as Exhibit 99.1 hereto and incorporated herein by reference is a presentation that Ocugen, Inc. will post on its website on March 13, 2023 and may use from time to time in presentations or discussions with investors, analysts, and other parties.
Item 9.01 Financial Statements and Exhibits.
The following exhibits are being filed herewith:
(d) Exhibits
| Exhibit No. | Document | |||||||
| 99.1 | ||||||||
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document). | |||||||
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SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
Date: March 13, 2023
| OCUGEN, INC. | ||||||||
| By: | /s/ Shankar Musunuri | |||||||
| Name: Shankar Musunuri | ||||||||
| Title: Chief Executive Officer and Chairman | ||||||||
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Courageous Innovation March 2023 NASDAQ: OCGN
Forward Looking Statements 2 This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are based on the beliefs and assumptions of Ocugen, Inc. and on information currently available to management. All statements contained in this presentation other than statements of historical fact are forward- looking statements. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Forward- looking statements that we make in this presentation are based on a combination of facts and factors currently known to us and speak only as of the date of this presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation whether as a result of new information, future events, or otherwise, after the date of this presentation.
We’re Here to Make an Impact Through Courageous Innovation 3 Mission: Developing cutting-edge innovations for people facing serious disease and conditions with a commitment to ensuring global market access Pioneering modifier gene therapy for inherited retinal diseases, as well as larger blindness diseases with unmet need Innovating a novel biologic to treat eye diseases that can lead to vision loss for millions of people Developing vaccines for flu & COVID-19 Pursuing Regenerative Cell Therapy to treat serious conditions like articular cartilage lesions
4 Pipeline Overview Asset/Program Indication Current Status Cell therapies (Regenerative Medicine) NeoCart® (Autologous chondrocyte-derived neocartilage) RMAT* Treatment of Articular Cartilage Defects in the Knee • Phase 3 clinical trial is planned for 1H 2024 Gene therapies OCU400 ** AAV-hNR2E3 Gene mutation-associated retinal degeneration* Retinitis pigmentosa (RP)--NR2E3 Mutation • Phase 1/2 • Completed dose escalation and established maximum tolerable dose (MTD) • Completed recruitment of RP Patients • Encouraging safety profile to date RP—RHO Mutation Leber congenital amaurosis (LCA)—CEP290 Mutation OCU410 AAV-hRORA Dry Age-Related Macular Degeneration (Dry AMD) • IND planned for 2Q 2023 OCU410ST AAV-hRORA Stargardt disease (orphan disease) Biologics OCU200 Transferrin – Tumstatin Diabetic Macular Edema • IND submitted in February 2023; preliminary results anticipated 4Q 2023 Diabetic Retinopathy • IND-ready Wet Age-Related Macular Degeneration (Wet AMD) • IND-ready Vaccines OCU500 Series OCU500: COVID-19 (Bivalent) For Prevention of Disease Caused by COVID-19 • IND planned for 4Q 2023 OCU510: Flu (Quadrivalent) For Prevention of Disease Caused by Flu OCU520: Flu + COVID-19 For Prevention of Diseases Caused by Flu and COVID-19 COVAXIN™ (BBV152) SARS-CoV-2 virus For Prevention of Disease Caused by COVID-19 • EUA for adults in Mexico • Phase 2/3 enrollment complete and top line results released o Final data and analysis anticipated mid-year 2023 *Regenerative Medicine Advanced Therapy Designation **Broad , gene-agnostic , ORPHAN DRUG DESIGNATIONS FOR RP/LCA FROM FDA AND EMA
5 NeoCart® (Autologous chondrocyte-derived neocartilage)
6 *The Journal of Bone & Joint Surgery: June 1, 2011 - Volume 93 - Issue 11 - p 994-1000 **https://www.biospace.com/article/cell-therapy-market-size-cagr-trends-forecast-report-2022-2030/ NeoCart is a regenerative cell therapy technology • Combines bioengineering and cell processing to enhance autologous cartilage repair • Potential to accelerate healing and reduce pain through reconstructing damaged knee cartilage High prevalence of knee cartilage damage, with progression to osteoarthritis (OA) • Arthroscopic knee procedures: over 1M annually* • OA: 528M diagnosed worldwide • Cell therapy global revenue forecast: $45B+, with North America expected to hold largest share** Current therapies to treat cartilage damage in the knee suboptimal • Varying outcomes due to variable cellular responses • Current standard of care suffers from one or more of the following: pain, reduced knee function, failure to address cartilage damage, donor tissue availability, open surgery NeoCart potentially addresses shortcomings of current treatments • Treat pain, improve function, and prevent progression to OA • Potential for improved efficacy, long-term benefits Program advancing on several fronts • Received FDA concurrence on confirmatory trial design of Phase 3 (initiate 1H 2024) • Renovating facility to accommodate cGMP manufacturing Follow-up Arthroscopy Demonstrates NeoCart® Progression and Integration** Initial Lesion Time Zero Implantation 8 Weeks 6 Months NeoCart®: U.S. FDA Agreed to Proposed Control and Overall Design for Phase 3 Trial to Evaluate Safety and Efficacy Compared to Chondroplasty Standard of Care
7 Modifier Gene Therapy Platform Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions
8 Modifier Gene Therapy: A Broader Reach Gene modifier therapy can potentially address multiple genetic defects with a single product utilizing a gene agnostic approach. In patients with IRDs, this could mean: Improved visual outcomes and quality of life GENE X GENE M cell NR2E3 Delivery “Molecular reset” of health & survival gene networks Cell with normal function cell Restored retinal cell homeostasis O N L ONL/photoreceptor survival GENE X cell GENE M Cell with mutated/ nonfunctioning gene(s) other than modifier gene M O D I F I E R G E N E M
FDA granted expanded Orphan Drug Designations for all retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) mutations Despite its prevalence, RP and LCA patients have limited treatment options • US: RP & LCA affect 110,000 and 15,000 people, respectively • Worldwide: conditions affect approximately 1.6M people Current approved and in-development gene therapies focus on individual gene • More than 125 mutated genes associated with RP and LCA • Developing a single therapy to treat each mutation is not feasible OCU400 addresses shortcomings of current gene therapy approaches • Broad-spectrum, gene-agnostic approach to genetically diverse inherited retinal diseases • Potential one-time, curative therapy with a single sub-retinal injection, using NR2E3 Dose escalation and recruitment of RP patients completed • High dose established as Maximum Tolerable Dose (MTD) • Continue to enroll patients with LCA • Intend to initiate a Phase 3 trial near the end of 2023 9 OCU400: Phase 1/2 Clinical Trial Progressing as Planned, Developing a Novel Gene Therapy in Ophthalmic Areas of High Unmet Need
A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa associated with NR2E3 and RHO mutations and Leber Congenital Amaurosis with mutation(s) in CEP290 gene Clinical Trials.gov Identifier: NCT05203939 Safety of subretinal administration of OCU400 Best Corrected Visual Acuity (BCVA) Multi-Luminance Mobility Test (MLMT) Full-Field Stimulus Test (FST) NEI-VFQ-25 Primary Endpoint: Safety Key Exploratory Efficacy Measures 10 OCU400-101 Clinical Program
11 OCU400: Expected Pathway to Clinical Development & Potential Approval RP LCA Proposed Indication RP & LCA Both FDA & EMA granted broad orphan drug designation for RP & LCA
Macular Degeneration Age related GA Drusen Deposit Old age STGD ABCA4 mutation Fatty Deposits Early Age Retina Degeneration Vision Loss Genetic Drusen Fatty Deposits 12 Dry AMD Limited options, presenting significant unmet medical need • US: 10M • Worldwide: condition affects more than 266M people Stargardt--an orphan disease No treatment options exist • US: 35,000 • Worldwide: condition affects approximately 800,000 people Recently approved therapy for geographic atrophy (GA)—advanced form of dAMD–has limitations • Frequent intravitreal injections (N ~6-12 doses per year); Patient compliance • Limited effect of GA lesion growth rate • Approximately 12% of patients experience neovascular AMD when the drug is administered every month for two years OCU410 addresses shortcomings of current approaches • Broad-spectrum, gene-agnostic approach • Potential one-time, curative therapy with a single sub-retinal injection, using RORA Plan to Initiate Phase 1/2 clinical trial in 2Q 2023 OCU410: Dry Age-related Macular Degeneration (dAMD) and Stargardt Disease (STGD)
Ap pr oa ch es b ei ng in ve st ig at ed Antioxidants Complement cascade inhibitors Neuroprotective Visual cycle inhibitors Gene therapy Cell-based therapies A strong role of inflammation, complement, and oxidative pathways in AMD Pegcetacoplan (Syfovre) - approved Lampalizumab Zimura sCD59 AREDS AREDS2 (failed) OT-551 (failed) Corticosteroids (repeated dose) 13 AMD: Risk Factors, Treatment Options and Unmet Needs
50 60 70 80 90 1e61E51E41E31E2Cells N or m al ize d Ce ll Su rv iv al RORA NR2E3 NR1D1 Anti-oxidative: Improves ARPE19 cells survival Anti-complement: Increased anti-compliment (Cd59) protein Anti-inflammatory: Suppresses inflammation in HMC3 cellsAnti-drusen activity and improves retinal function 0 50 100 150 200 B lu e A ut of lu or es ce nc e (A U) Months 1 7 1 7 1 7 Control Abca4-/- Uninjected Abca4-/- OCU410 ✱✱ ✱✱✱ Macular Degeneration Model Reduced Drusen 14 OCU410 (RORA): A Potential Modifier Therapeutic for Dry-AMD and STGD
15 OCU200 Novel biologic for treating Diabetic Macular Edema (DME), Diabetic Retinopathy (DR) and Wet Age-Related Macular Degeneration (Wet AMD)
16 OCU200 is our novel biologics candidate for sight-threatening conditions • A recombinant fusion protein of transferrin and tumstatin • Potential to address diabetic macular edema (DME), diabetic retinopathy (DR), wet AMD High prevalence of DME, DR and wet AMD patients • DME: 21M worldwide • DR: 162M worldwide • Wet AMD: 30M worldwide Limited treatment options available for the above patients • Current therapies target only one pathway, either angiogenesis or inflammation • Up to 50% of patient population are not responsive to current treatments OCU200 potentially addresses shortcomings of current treatments • Intended to target multiple causative pathways such as angiogenesis, oxidation, inflammation • Potential to offer better treatment options for all patients Company submitted an IND application on February 27, 2023 • Initially targeting DME Diabetic Macular Edema: bulges protrude from the blood vessels, leading to leakage of fluid and blood into the retina; leakage results in swelling (or “edema”), promoting vision loss. OCU200: Submitted an IND with the U.S. FDA to Initiate a Phase 1 Clinical Trial Targeting Diabetic Macular Edema (DME)
17 OCU500 Series: OCU500: COVID-19 Mucosal Vaccine OCU510: Flu OCU520: COVID-19/Flu
18 OCU500 A bivalent COVID-19 vaccine OCU510 A seasonal quadrivalent flu vaccine OCU520 A combination quadrivalent flu and bivalent COVID19 vaccine Next-Generation Vaccine Candidates Using Inhalation Technology to Potentially Overcome Durability and Transmission Challenges COVID-19 and flu infections continue to be a public health concern • COVID-19: 1M+ U.S. cases in the last 30 days; 5M+ WW cases with 47K deaths in the last 28 days • Flu: 50%+ of U.S. population 6-months and older received a shot for the 2022 to 2023 flu season, totaling 170M doses Limitations of current Flu vaccines • Sub-optimal vaccine efficacy (~20-40%); rapid decline in immune responses; poor local immunity at infection site • Manufacturing challenges for egg-based vaccines; antigenic drift during production- reducing vaccine efficacy Limitations of current COVID-19 vaccines • Lack of durability: immunity wanes significantly over time, requiring repeated boosters • Inability to stop transmission: breakthrough infections prevalent, increasing potential for mutations Inhalation vaccine advantages • Potential to generate rapid mucosal immunity in respiratory pathways, limiting infection and transmission • COVID-19 preclinical studies demonstrated vaccine induced high neutralizing and effector responses Antigenic Landscape Challenges and Opportunities: Flu and Covid-19 Vaccines
• Multiple RBD mutations > less cross-protection • Change in infection and pathogenesis pattern > reduced latency period • Reduce cellular memory > limited tissue-resident memory T-cells Limited local immunity at site of infection • Decrease in neutralizing IgG antibody levels • Lack of a robust secretory IgA response The rapid emergence of variants New SARS-CoV-2 variant frequencies in U.S. https://www.science.org/doi/10.1126/science.abm0620 Decline in Vaccine Efficacy 19 Factors Responsible for the Rapid Decline in Vaccine Efficacy
20 • Maximizing the area of vaccine delivery to the respiratory tract (Intranasal vs. Inhalation) • Formulations to facilitate the adsorption and prolong the retention time on respiratory epithelial cells • Delivery and uptake of antigens to target APC cells such as M-cells, DCs, and macrophages Enhancing mucosal vaccines Inhalation Intranasal Updated vaccine antigens • Introduction of bivalent / multivalent vaccines • Variant-modified COVID-19 vaccine boosters • QuadFluconstructs ( type A and B) • Induction of polymeric IgA in the respiratory tract prevents viral transmission • Prevent systemic spread by inducing high levels of circulating IgG titers • Mucosal vaccines proved in multiple animal models of infection and clinical trials Prevent both transmission and systemic spread with mucosal vaccines Strategies to Improve COVID-19 and Flu Vaccines
21 Approved (Ex-U.S.) Mucosal COVID-19 Vaccines: Demonstrated Safety & Efficacy as a Heterologous Booster Studies demonstrating the benefit of AAV Improved Immunogenicity in Ph3: iNCOVACC vs. COVAXIN • Superior GMT ratio of nAbfor Wuhan (1.45) • Superior GMT ratio of nAbfor OmicronBA.5 (2.1) • GMT ratio for secretory IgA in saliva (1.3) Improved Safety in Ph3: iNCOVACC vs. COVAXIN • Systemic AEs 2.7% (iNCOVACC) vs. 6.2% (COVAXIN) • Nasal reactions 4.9% (iNCOVACC) • Injection reactions 23% (COVAXIN) Ph 3 (N=2160): Superior Immune Response • iNCOVACC® (N=3000) vs. • COVAXIN™(N=160) CanSino Bio: Ad5-Nebulizer/InhaledBharat Biotech: ChAd-Nasal Dropper Five booster studies Ph 3 (SeiHOPE trial): N=13000 Dose : 1/5 of IM dose Improved Immunogenicity: • Cross protection against Omicron with heterologous booster • Produced T-cell responses higher than IM route • Significantly higher neutralizing antibody responses to WT and Omicron BA.1 compared with inactivated vaccine • Improved serum IgA antibody titers vs. inactivated and subunit vaccines for BA.4/5 Improved Safety: iNCOVACC vs. Inactivated Vaccine • Significantly lower number of injection site reactions vs. inactivated vaccine References: https://doi.org/10.1101/2022.03.08.22271816; https://doi.org/10.1101/2022.06.03.22275983; https://doi.org/10.1080/22221751.2022.2132881 https://doi.org/10.1080/22221751.2022.2155251 Ocugen using ChAd vector with inhalation technology for the mucosal vaccine platform (Flu, COVID-19 & Combo ) https://www.thelancet.com/journals/lanres/article/PIIS2213-2600(22)00087-X/fulltext https://www.thelancet.com/journals/lanres/article/PIIS2213-2600(23)00049-8/fulltext https://doi.org/10.1101/2022.07.26.22278072 https://ssrn.com/abstract=4000565 https://ssrn.com/abstract=4304747
22 COVAXIN™ (BBV152) A whole-virion inactivated COVID-19 vaccine candidate licensed from Bharat Biotech (BBIL) for North American Markets
23 COVAXIN™(BBV152): Final Data and Analysis Expected Mid-Year 2023 for Our Injectable COVID-19 Vaccine Enrollment completed for Phase 2/3 immuno-bridging and broadening clinical trial in December 2022 Topline data highlights, reported in January 2023, include the following: • Safety: well-tolerated with no related serious adverse events (no thrombotic, myocarditis, pericarditis cases) • Efficacy: immunogenicity demonstrated • Final data and analysis: anticipated mid-year 2023
24 Ocugen™ Vision Fully integrated, patient-centric biotech company focused on vaccines in support of public health and gene and cell therapies targeting unmet medical needs through Courageous Innovation
March 2023 NASDAQ: OCGN